Expanding Approved Patient Populations for Rare Disease Treatment Using In Vitro Data

In vitro cell‐based data can be used to support the extension of pharmaceutical approval to patient subsets with unique genetic variants. A set of conditions should be satisfied to support the extension of approval. The disease mechanism should be well described, and the impact of variants on protei...

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Detalles Bibliográficos
Autores principales: Weaver, James L., Wu, Wendy, Hyland, Paula L., Lim, Robert, Smpokou, Patroula, Pacanowski, Michael
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Reviews
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293129/
https://www.ncbi.nlm.nih.gov/pubmed/34496049
http://dx.doi.org/10.1002/cpt.2414

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9293129/
https://www.ncbi.nlm.nih.gov/pubmed/34496049
http://dx.doi.org/10.1002/cpt.2414

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